Health Trials

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

ConditionsPrimary Immune Deficiency DisorderImmune Deficiency DiseaseBone Marrow FailureShort Telomere LengthFanconi AnemiaNon Fanconi DNA-DSB Repair DisorderHoyeraal-Hreidarsson SyndromeDyskeratosis CongenitaTelomere Biology DisorderShort Telomere Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age4-50
SponsorSidney Kimmel Comprehensive Cancer Center at Johns Hopkins

About this trial

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.

Eligibility criteria

Qualifiers

Chronic granulomatous disease (CGD)

Wiskott-Aldrich syndrome (WAS)

Hyper-IgM syndrome

Common variable immunodeficiency (CVID)

Disqualifiers

Patients will not be excluded on the basis of sex, racial or ethnic background.

Positive leukocytotoxic crossmatch.

Prior allogeneic stem cell transplant.

Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment. The investigators recognize that patients with CAEBV may have ongoing EBV viremia at the time of initiating pre-transplant therapy, but other patients should have no uncontrolled bacterial, viral, or fungal infections.

Trial design

Treatments tested in this trial

  • Alemtuzumab
  • Fludarabine
  • Melphalan
  • Low Dose Total Body Irradiation
  • Cyclophosphamide
  • Tacrolimus
  • Mycophenolate Mofetil

Treatment groups

27 Participants
are divided into 3 treatment groups

Sponsors and collaborators

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Lead sponsor

Maryland Stem Cell Research Fund

Collaborator

Source ClinicalTrials.gov